Portfolio News

EINDHOVEN, the Netherlands — April 2, 2024 — ONWARD Medical N.V. (Euronext: ONWD), the medical technology company creating innovative spinal cord stimulation therapies to improve or restore movement, function, and independence in people with spinal cord injury (SCI), today announces it has submitted its De Novo application to the US Food and Drug Administration (FDA) to allow marketing of its breakthrough ARC-EX System to restore function of the upper extremities after SCI.

The submission marks an historic milestone for the Company in its mission to restore mobility and function for people with SCI. Once cleared by the FDA, ARC-EX will be the first-ever spinal cord stimulation therapy to restore hand and arm function after SCI and the first commercial product for ONWARD Medical. ONWARD prioritized upper limb function as its first indication for the ARC-EX System given feedback from the SCI Community of the importance of arm, hand, and finger function in empowering independence after SCI.

“We are delighted to be one step closer to bringing our breakthrough ARC-EX System to people living with SCI after submitting this De Novo application for regulatory clearance in the United States,” said ONWARD Medical CEO Dave Marver. “This therapy has the potential to transform the lives of people living with paralysis, while also positively impacting their loved ones.”

The De Novo application follows the Company’s global pivotal study – called Up-LIFT – the first large-scale pivotal study of transcutaneous spinal cord stimulation. The study investigated the safety and effectiveness of ARC-EX Therapy in improving upper limb strength and function in 65 study participants with chronic tetraplegia at 14 leading SCI neurorehabilitation centers in the United States, Canada, the United Kingdom, and the Netherlands. The study met all primary safety and effectiveness endpoints and demonstrated that 72% of participants responded to ARC-EX Therapy1, showing improvement both in strength and function.

“The SCI Community is eager to have access to this innovative technology,” said Candy Tefertiller, PT, DPT, PhD, NCS, Executive Director of Research and Evaluation, Craig Hospital in Lakewood, Colorado. “Even a small difference in hand and arm function can have a profound impact on independence and quality of life. The results of the Up-LIFT trial that led to this submission represent a significant advancement in the use of neuromodulation for individuals with spinal cord injury.”

The ONWARD ARC-EX System delivers ARC-EX Therapy™ – targeted, programmed electrical stimulation – transcutaneously to the spinal cord to increase strength, movement, and function of the upper limbs after SCI. The ARC-EX System was previously awarded FDA Breakthrough Device Designation (BDD) for upper limb function, which provided prioritized FDA review, the opportunity to interact with FDA experts, and the potential for additional reimbursement.

Nearly 200,000 people in the US and Europe have incomplete impaired upper extremity function after spinal cord injury.2,3 The Company is preparing for regulatory submission in Europe next.

To learn more about ONWARD Medical’s commitment to partnering with the SCI Community to develop innovative solutions for restoring movement, function, and independence after spinal cord injury, please visit ONWD.com.

*All ONWARD Medical devices and therapies, including but not limited to ARC-IM®, ARC-EX®, ARCBCI™, and ARC Therapy™, alone or in combination with a brain-computer interface (BCI), are investigational and not available for commercial use.

PHILADELPHIA, April 1, 2024 /PRNewswire/ — Carisma Therapeutics Inc. (Nasdaq: CARM) (“Carisma” or the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced the appointment of John Hohneker, M.D. to the Board of Directors of the Company, effective April 1, 2024. Additionally, the Company announced that Chidozie Ugwumba, who has served on Carisma’s Board of Directors since December 2020, has advised the Board of his intent to step down from his role as a member of the Board and chair of the Audit Committee of the Board effective April 1, 2024, as a result of his other professional commitments.

“We are pleased to welcome John to the Carisma Board of Directors,” said Sanford Zweifach, Chair of the Carisma Board of Directors. “I believe John’s extensive track record of clinical development, strategic leadership, and public company Board membership in the biopharmaceutical space will enable him to immediately support the Company as it continues leading the field of engineered macrophage development. We would also like to thank Chidozie for his invaluable advice and guidance since he first joined the Board over three years ago.”

“I am excited to join Carisma’s Board as the Company continues to develop innovative immunotherapies,” said Dr. Hohneker. “I am looking forward to partnering with the Board and the management team as they work to potentially bring novel treatments to patients in need across multiple therapeutic areas.”

Dr. Hohneker brings over 30 years of extensive experience in drug development and leadership across the biotech and pharmaceutical sectors. He most recently served as President and Chief Executive Officer of Anokion SA. Prior to this, he held the role of President of Research and Development at FORMA Therapeutics Inc. Before joining FORMA Therapeutics, Dr. Hohneker held roles of increasing responsibility at Novartis AG, including most recently as Senior Vice President and Global Head of Development, Immunology, and Dermatology. Prior to his time at Novartis, Dr. Hohneker held positions of increasing responsibility at Glaxo Wellcome and its legacy company, Burroughs Wellcome.

Dr. Hohneker currently serves on the Board of Directors of Curis, Inc., Sonata Therapeutics, Inc., and Trishula Therapeutics, Inc. Previously, Dr. Hohneker has served as a member of the Board of Directors of Torque Therapeutics, Inc., Dimension Therapeutics, Inc., Cygnal Therapeutics, Inc., BioTheryX Inc., Evelo Biosciences, Inc., Humanigen, Inc. and Aravive, Inc. Dr. Hohneker received his bachelor’s degree in chemistry from Gettysburg College and his M.D. from the Rutgers School of Biomedical and Health Sciences (formerly the University of Medicine and Dentistry of New Jersey-Rutgers Medical School). He completed his internship and residency in internal medicine and his fellowship in medical oncology, all at the University of North Carolina at Chapel Hill.

Berlin, Germany, March 14, 2024 – Bayer and Aignostics GmbH today announced a strategic collaboration on several artificial intelligence (AI)-powered approaches with applications in precision oncology drug research and development. Aignostics is a spin-off from one of the world’s leading hospitals, Charité-Universitätsmedizin Berlin, and a global leader in using computational pathology to transform complex biomedical data into biology insights.

The partners will co-create a novel target identification platform that leverages Aignostics’ technology and proprietary multimodal patient cohorts, and Bayer’s deep expertise in discovering and developing novel oncology therapies. In addition, the collaboration will include the development of computational pathology algorithms powered by AI and machine learning (ML) that connect baseline pathology data, such as molecular tumor profiles, with clinical data, such as patient outcomes, to enable better patient identification, stratification, and selection for clinical trials.

The goal of the multi-year research collaboration is to identify novel cancer targets with a strong disease link through AI models applied to multimodal patient data and to accelerate clinical development of oncology programs. This approach has the potential to address some of the challenges currently experienced in target discovery and disease heterogeneity.

“Gaining insights into human disease biology, discovering targets with a strong disease link by integrating artificial intelligence, machine learning and multimodal pathology into precision drug development has a huge potential for our R&D innovation strategy,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “Bringing Aignostics’ technical knowhow and their access to large patient datasets together with Bayer’s expertise in cancer research and development will enable discoveries and faster clinical development, helping to provide cancer patients with more impactful medicines.”

The collaboration will leverage Aignostics’ technology and access to longitudinal, multimodal clinical data sets in well characterized patient cohorts to discover new oncology targets for high unmet medical need indications. Under the terms of the agreement, the companies will collaborate on multiple discovery programs and initiate at least two target identification programs.

“Innovation at Aignostics has always been fueled by close collaboration with clinicians and biopharma. With this partnership, we’re thrilled to take that approach to the next level. Fusing our technology and multimodal data with Bayer’s extensive expertise in drug discovery and clinical development has the potential to generate better drugs for patients with high unmet need in less time. Together with Bayer, we’re excited to transform AI’s immense potential into a reality for healthcare,” said Viktor Matyas, CEO of Aignostics GmbH.

Aignostics will receive an upfront payment and is eligible to receive success-based milestone payments and royalties on any commercialized therapies that result from the collaboration.

Positive results demonstrate promising new option for women seeking better, personalized treatment

GRENOBLE, France and MINNEAPOLIS, Feb. 14, 2024 /PRNewswire/ — UroMems, a global company developing innovative, mechatronics technology to treat stress urinary incontinence (SUI), announced today that it has successfully met the six-month primary endpoint for the first-ever female patient implanted with the UroActive™ System, the first smart automated artificial urinary sphincter (AUS) to treat SUI. This milestone indicates a new era for millions of women suffering from SUI, and signals an exciting transition for surgeons treating SUI not only in France, where the female patient was treated, but also across Europe and the U.S. It also follows closely on the heels of the successful results of the complete treatment cohort of the first-in-man clinical feasibility study. Results of this clinical study will contribute to the design and implementation of UroMems’ pivotal clinical trial in Europe and the U.S.

“On behalf of the medical team who implanted the UroActive System in the first female patient, Drs. Christophe Vaessen, Aurelien Beaugerie and I are over the moon to see our first patient has returned to living life fully after years of struggling with SUI,” said Professor Emmanuel Chartier-Kastler. “This promising therapy is a breakthrough technology in treating SUI in both women and men.”

The primary outcome measures include the successful device activation and the rate of explants and revisions at six months. The first female patient has not only met the study’s primary endpoints by remaining revision-free but is also experiencing restored social continence. Follow-up on secondary measures, including leak rate values, has been extremely positive.

“As the leading advocacy organization in the U.S. supporting patients and caregivers with urinary incontinence, we hear from patients weekly looking for information on the safety and efficacy of artificial sphincters. Unfortunately, we have little to report back and support their requests as no one in the U.S. actively promotes this option for women,” said Steven Gregg, Ph.D., executive director of the National Association for Continence. “These results of the first UroActive System implanted in a female represent a promising development to treat stress urinary incontinence in both women and men. We’re excited about this first-of-its-kind development and look forward to UroMems’ pivotal trial results.”

Pending those results, the potential for U.S. surgeons to offer this new option is now on the horizon; skilled surgeons performing robotic-assisted surgeries such as sacrocolpopexies and hysterectomies may soon be able to add implanting UroActive to their standard practices. UroActive is the first smart active implant that treats SUI, powered by a MyoElectroMechanical System (MEMS). This innovative system is placed around the urethra in men and the bladder neck in women, controlled based on the patient’s activity, without the need for manual adjustments, intending to provide patients with ease of use and a better quality of life than current options.

“We are elated to reach this critical achievement contributing to the demonstration of the feasibility of the UroActive System to successfully treat women suffering from debilitating SUI,” said Hamid Lamraoui, UroMems chief executive officer and co-founder. “The compelling results of this first-in-female implant show the high potential of our technology, bringing us one step closer to delivering on the massive unmet need for women and physicians desperately seeking a better SUI treatment option.”

SUI, or involuntary urinary leakage, affects an estimated 40 million Americans and 90 million Europeans, and occurs when the pressure in the bladder exceeds that of the muscle (the sphincter) around the urethra, caused by activities involving high intra-abdominal pressure, like coughing, laughing and exercising. SUI significantly impacts quality of life, as it can be debilitating, and often leads to depression, low self-esteem and social stigma.

UroMems aims to restore the quality of life, dignity and self-esteem of millions of men and women worldwide suffering from poorly treated chronic conditions by the commitment to change the perception that these disorders are inevitable as one grows older and is simply something to endure with no real solution. UroMems is revolutionizing the treatment of SUI with smart active implants, using the latest technological advances in the field of embedded systems and micro-technologies for the development of its groundbreaking solutions.

Cambridge, Mass. (January 18, 2023) – eGenesis, a biotechnology company developing human-compatible organs and cells for the treatment of organ failure, and OrganOx, a medical device company with a focus on the therapeutic applications of isolated organ perfusion, today announced the successful completion of an extracorporeal perfusion of a brain-dead research donor using a genetically engineered porcine liver. The donor was the first to be enrolled in the ongoing PERFUSE-2 study.

The study, made possible through the generosity of a donor family seeking to help other families through the advancement of clinical research, was carried out in collaboration with the University of Pennsylvania Transplant Institute and Gift of Life Donor Program. The perfusion was performed using the eGenesis liver, EGEN-5784, connected to the OrganOx extracorporeal liver cross-circulation (ELC) device to enable circulation of the donor’s blood through the porcine liver. Stable blood flow, pressure, and pH were achieved throughout the procedure in addition to robust bile production. No evidence of rejection was observed. The perfusion was electively stopped per-protocol at 72 hours with the liver appearing healthy.

The PERFUSE-2 study is being conducted to evaluate the feasibility of using this liver perfusion system to support patients suffering from liver failure. Annually in the US, over 300,000 patients are admitted with various forms of liver failure, requiring treatment in the acute setting. The efficacy of existing liver support options is limited, and patients in liver failure face a high risk of mortality. For some patients, utilizing a human-compatible, genetically engineered porcine whole liver to support the function of a patient’s decompensated liver may provide time for the recovery of the patient’s native liver or time to obtain a liver transplant. eGenesis and OrganOx are co-developing this technology and anticipate submitting an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration (FDA) in 2024 to initiate a first-in-human clinical study.

The genetically engineered porcine liver used in this study carried the same genetics as the porcine kidneys used in the landmark preclinical study recently published in Nature. These edits include (1) knock out of three genes involved in the synthesis of glycan antigens implicated in hyperacute rejection, (2) insertion of seven human transgenes involved in the regulation of several pathways that modulate rejection: inflammation, innate immunity, coagulation, and complement, and (3) inactivation of the endogenous retroviruses in the porcine genome.

“We wish to extend our deepest gratitude to the donor and their family for enabling this important medical achievement and for paving the way for future work toward a potential solution for the many patients in need of life-saving liver support,” said Michael Curtis, Ph.D., President and Chief Executive Officer of eGenesis. “In addition to being a first in the field of xenotransplantation, this study provides important information to help advance our IND application.”

Abraham Shaked, M.D., Ph.D. of the University of Pennsylvania Transplant Institute said, “The success of this study provides support for further exploration of organ products developed using advanced genome engineering to provide novel, high-quality therapeutic options for individuals experiencing organ failure.”

“This marks a key milestone in our journey towards an effective treatment for acute liver decompensation. The OrganOx ELC system combined with a genetically engineered liver from eGenesis links modern organ perfusion technology with the functions of a whole liver, with the goal of providing a treatment that offers a lifeline to the critically ill patient – time for their own liver to recover or time to receive a transplant,” said Prof Peter Friend, Chief Medical Officer, OrganOx.

“Gift of Life Donor Program, one of the nation’s leading organ procurement organizations, is proud to have collaborated on this pioneering study that has the potential to bring new hope to waitlist patients by providing a bridge to transplant or time for healing,” said Richard D. Hasz, Jr., MFS, CPTC, President and CEO, Gift of Life Donor Program. “This unique study was only possible thanks to the generosity of a donor family willing to help alleviate the suffering of others despite their own personal loss. Every day, we are inspired by the kindness of families who choose donation. We look forward to partnering with Penn Medicine, eGenesis and OrganOx on future trials to advance this evolving field of medicine.”

“Our family is very proud to support this medical advancement and see our loved one’s legacy benefit countless others,” said a member of the donor family. “It is a testament to our loved one’s selflessness and compassion to know this donation offers such hope for people suffering serious disease in the future.”

PHILADELPHIA, Dec. 14, 2023 /PRNewswire/ — Carisma Therapeutics Inc. (Nasdaq: CARM) (“Carisma” or the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced the nomination of its first lead candidate under the collaboration with Moderna, Inc. (Nasdaq: MRNA). This first lead candidate will target an antigen present on a solid tumor with significant unmet medical need. This strategic collaboration brings together Carisma’s chimeric antigen receptor macrophage (CAR-M) platform with Moderna’s messenger RNA (mRNA) and lipid nanoparticle (LNP) technologies to generate and develop in vivo CAR-M therapeutics for oncology.

“Following the compelling pre-clinical proof of concept data shared at SITC 2023, we believe in vivo CAR-M therapeutics that utilize Moderna’s mRNA/LNPs have the potential to benefit patients with a broad variety of cancers,” stated Michael Klichinsky, PharmD, PhD, Co-Founder and Chief Scientific Officer at Carisma. “The delivery of this first candidate demonstrates our ability to create novel in vivo CAR therapies that can be advanced toward the clinic. We are proud of the significant contributions made by both of the scientific teams at Carisma and Moderna towards this exciting program. We look forward to completing IND-enabling studies with the lead candidate and are excited about the prospect of bringing this therapy forward for patients with advanced solid tumors together with Moderna.”

Lin Guey, PhD, Chief Scientific Officer of Therapeutics Research Ventures and Biotherapeutics at Moderna, stated, “We are excited with the progress we’ve made to advance in vivo cell therapy (CAR-M) in collaboration with Carisma. Combining Carisma’s deep expertise in myeloid cell biology with our mRNA/LNP platform has allowed us to quickly advance the first lead candidate and we look forward to furthering its development, along with our continued collaboration with Carisma to develop novel therapies to treat patients.”

Pre-clinical proof of concept data were recently presented at SITC 2023, demonstrating the feasibility, efficacy, and tolerability of the in vivo CAR-M platform that utilizes Moderna’s optimized mRNA encapsulated in LNPs and is designed to redirect endogenous myeloid cells to exert targeted anti-tumor activity. The highlighted data demonstrated that Carisma’s CAR-M therapy can be directly produced in vivo, or within the body, and can successfully redirect endogenous myeloid cells against tumor-associated antigens using Moderna’ mRNA/LNPs. This novel approach to cancer immunotherapy offers an off-the-shelf solution that has the potential to increase access to CAR-based therapies and to be the basis of any CAR-M programs discovered or developed under the Carisma and Moderna collaboration.

Successful primary and secondary results demonstrate proof of feasibility in male patients, paving way for launch of large-scale pivotal clinical study in the U.S. and Europe

GRENOBLE, France and MINNEAPOLIS, Dec. 13, 2023 /PRNewswire/ — UroMems, a global company developing innovative, mechatronics technology to treat stress urinary incontinence (SUI), announced today it has reached a significant milestone: the complete treatment cohort in the first-of-its-kind clinical feasibility study has successfully reached the six-month primary endpoints.

The feasibility assessment of the UroActive System was completed through a prospective multicenter clinical study. UroActive is the first smart automated artificial urinary sphincter (AUS) to treat SUI, and the only one to reach this critical milestone. The results of this initial clinical study support design and implementation of UroMems’ pivotal SUI trial in Europe and the U.S. All six men are now implanted for at least seven months and up to fifteen months, with their devices operating as expected and no need for revision nor explant. In addition, extremely positive follow-up was received on secondary outcomes measures, including leak rate values and patient quality of life questionnaires.

UroMems received very positive feedback from all patients participating in the study cohort. “You have changed my life,” stated one of the study participants. “I can do all activities again, without stress, without anxiety!”

“We’re so pleased to see that our expectations about our device’s performance were met or even exceeded and delighted to successfully treat and receive such high praise from patients who had been suffering from SUI for years,” said Professor Pierre Mozer, UroMems chief medical officer and co-founder. “The results of this study will allow us to prepare our pivotal study which will be a major step in the development of UroActive.”

UroActive is the first smart active implant that treats SUI, powered by a MyoElectroMechanical System (MEMS). This innovative system is placed around the urethral duct and is controlled based on the patient’s activity, without the need for manual adjustments, intending to provide patients with ease of use and a better quality of life than current options.

“The very compelling results of this first-in-man clinical study demonstrate the high potential of our technology and pave the way for larger clinical trials that will allow us to demonstrate all the benefits we are expecting to offer patients suffering from debilitating SUI,” said Hamid Lamraoui, UroMems chief executive officer and co-founder. “We could not have reached this important milestone without the enthusiastic participation of the men in this initial study cohort. We’re extremely grateful to them for being a vital part in bringing this potentially revolutionary treatment to market.”

SUI, or involuntary urinary leakage, affects an estimated 40 million Americans and 90 million Europeans, and occurs when the pressure in the bladder exceeds that of the muscle (the sphincter) around the urethra, caused by activities involving high intra-abdominal pressure, like coughing, laughing and exercising. SUI significantly impacts quality of life, as it can be debilitating, and often leads to depression, low self-esteem and social stigma.

EINDHOVEN, the Netherlands — November 06, 2023 — ONWARD Medical N.V. (Euronext: ONWD), the medical technology company creating innovative spinal cord stimulation therapies to restore movement, function, and independence in people with spinal cord injury (SCI), today announces a publication in Nature Medicine using investigational ONWARD ARC Therapy to address gait challenges related to Parkinson’s disease.

The study participant described in the Nature Medicine publication has been living with Parkinson’s disease for nearly three decades. He has a severe gait disorder that has not responded to conventional therapies.

“I could hardly walk without frequent falls, several times a day. In certain situations, like getting into an elevator, I would stomp, freeze as they say,” said the study participant in a press release issued by Switzerland’s Lausanne University Hospital (CHUV).

In 2021, researchers from the Swiss Federal Institute of Technology (EPFL) and CHUV investigated the possibility that ONWARD ARC Therapy (precise, targeted electrical stimulation of the spinal cord) could address common side effects of Parkinson’s disease that negatively impact mobility. The team collaborated with Dr. Erwan Bezard, a renowned neuroscientist from France’s National Institute of Health and Medical Research (INSERM).

After the introduction of ARC Therapy and benefitting from a few weeks of rehabilitation, the study participant was able to walk without previously noticeable gait interruptions. Today, he uses ARC Therapy eight hours per day.

“I turn on the stimulation in the morning and turn it off in the evening. It allows me to walk better, to stabilize myself. Even stairs don’t scare me anymore. Every Sunday I go to the lake, and I walk about six kilometers. It’s awesome,” said the participant in the CHUV release.

“The breakthrough reported in Nature Medicine shows the remarkable potential to use the same technology platform and therapy we are developing for spinal cord injury to also address mobilitychallenges stemming from Parkinson’s disease,” says Dave Marver, CEO of ONWARD.

“It is impressive to see that by electrically stimulating the spinal cord the same way we have done in paraplegic patients, we may be able to address gait disorders due to Parkinson’s disease,” adds neurosurgeon Jocelyne Bloch, co-director with Professor G. Courtine of ONWARD research partner .NeuroRestore.

.NeuroRestore was awarded a $1 million grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to implant the ARC-IM System and investigate the effect of ARC Therapy in six additional participants with Parkinson’s disease. This study will assist ONWARD in determining whether to conduct additional clinical trials and potentially commercialize ARC Therapy in the future for those living with Parkinson’s disease.

“MJFF is committed to fulfilling the unmet needs of people living with Parkinson’s disease by ensuring the development of improved therapies,” said Katharina Klapper, Director of Clinical Research, MJFF. “The Foundation is pleased to award a grant for scientists at EPFL and ONWARD Medical to investigate the effects of ARC Therapy in people with experiencing gait challenges.”

All ONWARD devices and therapies, including but not limited to ARC-IM, ARC-EX, and ARC Therapy, are investigational and not available for commercial use.

Note: ONWARD has previously disclosed achievement of human proof-of-concept for Parkinson’s disease in its Company Presentation.